Current Edition

How Engaging Patients in Trial Design Maximises Orphan Drug Success

Orphan drug discovery presents both opportunities and challenges, with research in this space having some of the highest hurdles in medicine. Clinical trial design and execution require scientific expertise, operational acumen, regulatory knowledge and patient engagement skills, due to orphan drugs usually being used to treat small, complex patient populations. Barbara Zupancic and Michael Murphy of Worldwide discuss further, concluding with how to capture all the benefits of orphan trials – in particular, partnering with a CRO experienced with innovative approaches to orphan drug trials.